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Dr. Collins 11, Us 5, Harkin 5, Ms. Unser 4, Vitro 3, Fertilization 3, Dr. Nelson 3, Dr. Daly 3, Michigan 2, Washington 2, Vitro Fertilization 2, Diabetes 2, Wayne State University 2, U.s. 2, Christopher Reeve 2, Dr. Morris 2, Corneal Blindness 1, Dr. Pa Doocy 1, Leukemia 1, Anemia 1,
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  CSPAN    Today in Washington    News/Business. News.  

    September 17, 2010
    6:00 - 7:00am EDT  

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before chief judge walker in san francisco on the issue of gay marriage, and the supreme court intervened to stop televising on closed-circuit television. so the legislature, the congress, had better get busy and had better act on this subject so we do not await court action. put pressure on knowing what may or may not happen. i have a couple of questions for you, dr. collins. the first question relates to the impact of the judicial decision. and i have gotten information that more than $500 million has been expended on embryonic stem
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cell research -- well, actually, three questions. question number one is, what has the impact been on the scientists now using n.i.h. funding for embryonic stem cell research in terms of the uncertainty of the future? number two, what results have been taken in a positive sense, which i know are very good for the more than $500 million already expended? and what has been the consequence of the $10 million in the stimulus package where you informally told me that it has created the tremendous excitement and a new wave of enthusiasm by researchers who had been discouraged by the failure of congress to keep the pace, which we have moved from $12 billion to $30 billion, but failure to keep the pace in
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funding since 2003? >> senator, thank you for the question. and let me first say how appreciative i am personally and everyone at n.i.h. is for the strong leadership you have shown over these years in your advocacy for the value of medical research, and especially because we're talking about it today for stem cell research, that has been much appreciated, and your articulation of the importance has always been right on target, as it just was here in your opening statement. we are all grateful indeed for the way that you have shown that leadership. and you, together with our chairman, have played such a significant role in n.i.h. being at this exciting place that we are right now in terms of medical research opportunities that frankly, i didn't dream we would be at 10 years ago. but we also are here with this cloud over the enterprise in this very specific area of embryonic stem cell research. when the judge issued that preliminary injunction we were stunned and basically after
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interpretations by the department of justice took steps that we felt we had to with intramural researchers, who are working with federal funds at that very time, doing embryonic stem cell research, we had to ask them to stop. with extramural grantees, if they had already received a grant and were spending down the dollars that they had already been allocated, they could continue, but they would need to come back for a renewal on an annual basis. and we basically said within a year there will be no more funds because those annual renewals cannot be adhered to. and frankly, we had a bunch of new grants and renewals right in front of us -- about 244 grants, adding up to about $200 million -- that were immediately put on hold, not to mention a whole other set of grants that were ready for peer review that we had to stick on the shelf because we felt that the judge's order prevented us from acting on them. fortunately and to our great relief, although temporary
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relief it apparently is, the stay on that particular injunction last week allowed us to catch up and to go back to doing what we had been doing along, and we are working vigorously to be sure that we are doing the right thing here in terms of supporting the research that we always had intended to. but there is this cloud of uncertainty that hangs over the situation because of not a clear path forward. and i think as you will hear from others at this hearing, that is creating great anxiety, particularly among other young scientists, who wonder, do i have a career path here or is this something i better not get involved in because it's too uncertain? so the impact so far has been quite significant and is uncertain going forward. we are, as i tried to show in the graph in the opening statement, spending in the neighborhood of $188 million -- sorry, $138 million on
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non-embryonic -- i'm sorry, on embryonic and other types of non-adult -- let me try that again. we are spending $138 million on human embryonic stem cell research and all of that was put into jeopardy. and that's an estimate for fy-10. in terms of your question about the era dollars that have flowed to n.i.h., that has been an enormous infusion of energy and capability and excitement in a community that had been frankly struggling after five years of flat budgets. and many innovative ideas gogolaking for support. that infusion -- going lacking for support. that infusion energized a whole host of projects. one of my jobs is to read a lot of the grants that came in and it is some of the most exciting science that you can imagine, and we have used it specifically to encourage people to put forward
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out-of-the-box ideas that otherwise might not have seemed worth trying in a tough budget. this is breakthroughs in cancer, heart disease, diabetes, autism, things that really have changed the whole landscape because of this opportunity to empower the community in ways that they had not previously been able to do. and i want to thank you for your remarkable leadership in making that possible. of course, we have another anxiety there that the two years of the recovery act are coming to a close, and the momentum that was started is now somewhat in question -- >> if you would just talk about the advances generally, but specifically on stem cells with the $500 million expended. tell about the big results there. >> mr. chairman, before dr. collins answers that, a vote's been called and i want to go to the floor. so i want to say really quickly -- >> i understand, if i can deal with this really quickly. >> doctor, your testimony is compelling, and i really
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appreciate it. it clarified a lot face-off me. i want to thank senator harkin for his leadership but i want to thank senator specter, too, and we'll continue to carry your spirit forward on this critical issue. i just wanted to say thank you very much. we do have a vote and i want to make sure we get to the rest of the panel, so thank you. >> thank you. >> well, just focus for a moment, if you would, on the $500 million already expended on embryonic stem cell research and what tremendous advances have been made there. >> so that's a long list. it has given us the opportunity at the basic level to begin to understand what it is that takes this cell with all of this potential and triggers it to become a neuron or a muscle cell or a pancreatic beta cell that makes insulin. those signals, that elaborate pathway of development, are now being sorted out by researchers
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with very powerful technology, some of them coming from the genome project. in terms of specific applications, you have heard of the application to spinal cord injury, which is now in its phase one clinical trial. that's the first one, which has actually made it through that. that was a lot of f.d.a. review, believe me. but there are also applications which are looking very promising for eye diseases and for type one diabetes, where human embryonic stem cells have been differentiated and then been used in an animal model to show clear benefit and rigorous science, setting the stage, then, for human clinical trials in the not too distant future. on top of all that, human embryonic stem cells are being used to do drug screening. because if you're looking for a drug that might help somebody with a muscle disease, you'd really like to test and see, does that work in human muscle cells? we now have the ability to make human muscle cells because you can take human embryonic muscle cells and tweak them to do
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that, and then test hundreds of thousands of compounds and find out what's there to stabilize this disease and make it better able to survive. a huge opportunity in drug screening, which is happening both in the private sector and in academia. all of those things add up to that roughly $500 and some million dollars, but we think we're just scratching the surface. >> thank you, dr. collins. that's powerful. >> thanks, senator harkin. dr. collins and others, there's two votes. we have about six or seven minutes left in this vote. so i'll recess the panel. we'll go vote on one and then we'll vote on the next one. so it will be probably 15 minutes before we get back here. so i'd like to see if anybody needs to use the facilities or something. we'll be back in 15 minutes. what i'd like to ask -- dr. collins, i hate to impose on you, but we're just -- there's a lot of things we need to cover.
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i'd like to ask you'd stay. while we're gone i'd ask the guards to put the nameplates of the second panel up there. but i do have follow-up questions for you, dr. collins, when we come back. >> no guards needed. i'm happy to stay. >> thank you, dr. collins. we'll be right back. host: caller[captioning performy national captioning institute] [captions copyright national cable satellite corp. 2010]
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>> the subcommittee will resume its sitting. dr. collins, two other things i wanted to cover with you. one, in your testimony -- and you mentioned it also in the slides -- was the power of embryonic stem cells as a research tool. sounds like even if these cells never actually end up being used as therapies in which they are tnsmitted into human beings, they can still teach us valuable information that can lead to treatments and cures. i just want to ask, is that correct? and just a slight elaboration on that. >> that is correct. and in two ways. one is that human embryonic stem cells, because they represent that most potent, most undifferentiated cell
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type, but can be encouraged to go down various pathways to become brain cells or blood cells or muscle cells, they give us a window into how that development hpens in humans in a way that we didn't have before. and, again, if you are able to understand what those signals are, you can also infer what goes wrong if one of those signals misfires, and many birth defects and genetic diseases are in that category. the second way is the ability to use these cls, particularly if you turn them into neurons or muscle cells or whatever it is that you need to study most to screen for new dg therapies. the way we get drugs in the past has been involving a variety of approaches, trying to identify a small molece d organic compound that would have the right properties to do something you wanted to, but you've often had to try that in an animal model. these are human cells, and they're human cells that you can convince to behave pretty ch the way they would as a
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person, except they're there in the dish, so you can do it without the risks of toxicity. very powerful new way to feigned the next generation of -- find the next generation of drugs. >> dr. collins, this is more on a personal note. opponents of human embryonic stem cell research sometimes argue that it's immoral. they've raised it into a moral issue. quite frankly, have a numbe of friends, but i have one very close family friend who had a lot of trouble conceiving a child. she and her husband tried many different things. they finally went to a fertility clinic and to in vitro fertilization. she was able to conceive and have a wonderful child. actually, twins. they are very healthy. and so that's a real blessing that science was able to help
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them. now, i don't know this for a fact, but in many of these cases a lot of the embryos are left over from a process of in vitro fertilization, and at some point the donors of asked what they want to do with them. obviously, they're not going to keep them in liquid nitrogen forever. so they're discarded, to which sometime ago my friend said to me, well, but i understand they could be used for embryonic stem cell research that might help someone who is suffering. and i said, well, yes, that's true. i would much rather do that. so seems to me there's some morality there that we haven't thought about. and as you know, under the
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guidelines that were issued, only stem cells derived from leftover in vitro fertilization can be used, with full consent of the donors and with no monetary consideration, and could not be transplanted into a uterus. had to only be used for stem cell derivation. so those are the ethical guidelines. i thought i'd lay that out. a lot of people don't understand that. but you are well known not only as one of the world's most well known scientists but as a man of faith. i actually did read your book. i thought it was very good. "the language of god." i think it's one of the wonderful crossover books between science and faith. it's a wonderful book. can you talk about why you personally, as a pre-eminent scientist, are comfortable with this research? how do you reconcile your advocacy for embryonic stem cell research with your own
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faith and your own mal judgment? >> thank you, senator. i think you've already articulated the issues extremely well. i do believe that the human embryo deserves moral respect. it is a potential human being, this coming together of sperm and egg. it's the way we all got started. and that is not something to be taken lightly. but when you lookt the circumstances that you ha just outlined in terms of the consequences of in vitro fertilization efforts, benevolent as they are, giving couples a chance to have children who otherwise could not, one of those consequences is the existence of hundreds of thounds of frozen elm b yoss and others that are -- embryos and others being discarded potentially all the time. and then faced with the ethical choice in that situation, i have come to the conclusion as a person of faith that the alternative of discarding this embryo that's clearly not going to get used versus, for a small
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number of these, trying to turn them into a stem cel line that might ultimately teach us something about human development and medicine and ultimately help us come up with a treatment for parkinson's disease or diabetes or spinal cord injury or some eye disease or liver disease. which of those is the more ethical choice? i think it's too easy to simply say, well, the embryo is an entity that is a potential human and, therefore, any consideration of using the word "research" in the same sentence is something we should be opposed to. we aren't really being given that as an alternative. these embryos exist. they're going to continue to exist as long as in vitro fertilization goes forward, and it is. certainly it's given many couples a chanceor a new life and their families. so putting the reality test here, i believe that most people who look carefully at the issues, whether from a faith perspective or a purely
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humanistic perspective, come up with a conclusion that what is potential here justifies what we are talking about in terms of fedel funding of human embryonic stem cell research. >> well, thank you very much for that profound statement. thank you very much, dr. collins. now our panel -- i'll introduce our panel here, and then we'll have our testimonies. dr. george daly, professor of hematology and oncology at children's hospital in boston and the dana far ver institute, also at harvard medical school. dr. daly is past psident of the international society for stem cell research and chaired the international task force that wrote ethical guidelines for human embryonic stem cell research. he received his ph.d. from m.i.t. and his m.d. degree from harvard medical school. dr. shawn morris, director of the university of michigan for
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stem cell biology, is also a professor of immediate sib and a professor in the life iences institute. es he also a director of the international society for stem cell research. received his ph.d. in immunolo from stanford university. gene nelson is an prosht professor at the department of aanatomy at wayne state university school of medicine. dr. nelson received her b.s. from the university of michigan, her ph.d. from wayne state university. and ms. unser is the founder of the first step foundation, an organization dedicated to raising research funds and public awareness for people afflicted with spinal cord-related paralysis. ms. unser graduated in may with a degree in biopolitics and is now a graduate student at the george washington university school of public health studying health poly. i thank you all for being here today. i thank you for your indulgenc
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because of our votes. i will make sure that all of your statements are made a part of the record in their entirety, and starting with you, dr. daly, working down, if you could sum up in five minutes or so -- i won't hold you to an exact time, b we'll start with five and try to get there so we can open it up for some discussion and question. again, dr. daly, no stranger to this subcommittee. welcome back. >> thank you very much. chairman harkin, thank you for the invitation to testify. i am here to assert that human cells offer unique advantages for understanding human diseases and are essential to a vigorous national portfolio of stem cell research. however, recent upheavals in the federal funding are disrupting our research and dissuading scientists from entering the field and threatening american pre-eminence in the research. as director of the stem cell transpla program at children's, i wish to first speak to the succe we have in using adult stem cells.
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and we're using adult stem cells to cure kids with a variety of life-threatening diseases. we perform some 80 stem cell transplants per year for childhood leukemia, genetic diseases, and indeed, we have cured many kids. i was on rounds last week the i met an adorable little girl about to receive her transplant for a very rare genetic immune disorder. and i found out she was the second in her family that we could very confidently say we would cure. so it's very, very heartening to save the life of a child. but i'm also here to advocate as a scientist. and as a scientist, i'm sobered by the statistic that fewer than half of all patients treated with stem cells are cured. and despite 50 years of research in adult stem cell transplants and practice, this is our most successful. blood cancers still relapse and patients still die. so as a scientist i'm working to improve these treatments through research on adult stem
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cells, embryonic stem cells and induced potent stem cells. i think it's a flawed argument to say that scientists should restrict their focus to adult stem cells and it's a mistake to cast the different types of stem cells as competing priorities. adult stem cells are not better than or more promising than embryonic stem cells. embryonic stem cells are different and to many scientists they offer more hope in certain diseases like diabetes. would it make sense as a federal policy to fund cancer and cord ya vascular research but not diabetes research? all of these are essential, and the most successful strategy to advancing cures is to let scientists decide which cells to study. now, i've been a student of the adult stem cell for 25 years, but starting about 15 years ago i began envisioning a new approach to the research to generating blood stem cells from embryonic cell cells. anthe idea was that we could
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generate customized blood stem cells in a way that would solve the immune rejection problem and the shortages and allow us to perform gene repair, together with bone marrow transplantation. now, we've succeeded in mice and we have a lot of promise in humans. in 2007 i was one of three laboratories to produce induced plurey potent stem cells and in 2008 my lab produced the first large repository of human disease-specific i.p.s. cells. so why, given that i have pioneered the development of both adult stem cells and induced potent stem cells do i continue to advocate for human embryonic stem cel? well, there's several reasons, and the first is that i.p.s. cells and many other future areas of research are founded on the knowledge we have gained from human embryonic stem cells. second, my own research and that of others is pointing to
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important differences between i.p.s. and e.s. cells. and third, some diseases are simply more effectively modeled with human cells than. i p.s. cells. we showed that you can model human anemia, a disease that pre-disposes kids to leukemia, as well as fragile x syndrome, which is the most common genetic cause of autism and mental retardation and these were better modeled with human embryonic stemroils. while we have so much to learn, how can we conclude that we don't need to fund the research? we're told that restrictions on federal funding won't inhibit stem cell research and that private philanthropy will fill the gap, but realistically research careers are founded on the architect of federal support. investment by the n.i.h. has made u.s. research pre-eminent. it's given us domination in the nobel pris and it's been an engine for our very vigorous biotechnology industry. now, opponents of e.s.l.
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research will argue that adult stem cells are more promising. that embryonic stem cells have yet to cure anyone. this is like arguing why try to develop new classes of antibiotics when we've got b sill lins and let's continue to improve those. it's very curious. the only time i confront the argument that adult stem cells are superior and that embryonic stem cells should be replaced is at hearings like this. at scientific meetings, we discuss and debate adult and embryonic and induced pro tent stem cells as all complementary aspects of cell and developmental biology. in my opinion, the arguments that adult stem cells obviate the need for embryonic stem cells are not scientifically driven. they are etiologically driven arguments to suppress e.s.l. research. and no matter how much progress is made with other forms of stem cells. e.s. cells will remain a vital
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research tool. e.s. cells are not contestants on "survivor" that should be voted off the island. expelling these cells will gravely weaken the search for cures. president obama's policy has expanded as sess to more e.s. cell lines and the court challenge has really come on us as a major blow. we've had immediate disruptions, but the long-term uncertainty is even more insidious. and i have several trainees who have toiled to make their projects on human e.s. cells work and the uncertainty has really compelled some of them to abandon those plans. so these decisions, which are driven by politics and not science, are deeply disturbing. so let me finish by saying that although the injunction has been stayed with the latest upheavals, we're again reminded that human e.s. cells are fragile and new legislation is needed to sustain the mo t tum of e.s. cell research and to allow scientists and not
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politicis nor judges to determine which research priorities to pure. thank you. >> dr. daley, once again, thank you for alhave profound statement and for all the work that you've been doing in this area. now we turn to dr. morris. dr. morris, welcome. >> thank you, senator harkin, for the opportunity to testify today. i've spent my entire career doing stem cell research, almost exclusively adult stem cell research. the research in my lab has won a number of awards, including an award from president bush in 2003. nonetheless, like nearly all leading stem cell researchers, i believe the federal government must support all forms of stem cell research, including human embryonic stem cells. we simply do not yet know what kinds of stem cells will yield the breakthroughs of the future and must pursue all forms of stem cell research to develop new therapies sooner, rather than later. stem cell scientists don't cluster into adult versus emyonic camps. this framing of the debate
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comes fro political lobbyists. i interact regularly with hundreds of the leading stem cell scientists throughout the world and virtually all of them believthat research must continue with all types of stem cells for the reasons george just articulated. stem cell research is a remarkably fast-moving field that has taken a series of unexpected twists and turns over the past several years. there's no point in the last 10 years wre we could have predicted, even two years down the road, where the field would be. yet, at every point the have been people who believe they could predict the future and who could tell us which avenues of research should be abandoned. but until the research is done, we don't know what the answers will be. think about the arguments that opponents have made as alternatives to embryonic stem cell research. fit they suggest that umbilical cord stem cells could replace embryonic stem cells. i can tell you that there was any any scientific plausible
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basis for the argument that cord stem cells could do what embryonic stem cells can do. instead, they subsequently suggested that amniotic cells could replace embryonic stem cells, but those cells are biologically different from embryonic stem cells and were never a plausible alternative. again, you never hear about those cells anymore. then opponents circulated lists of ove 70 diseases they claim could be cured with adult stem cells. what they don't tell you is that only diseases of the blood-forming system are routinely treated with adult stem cells and many of the other treatments, in quotation marks, they cite are highly specative are often not based on sound science and are prohibited from being sold to patients in this country by our food and drug administration. the reality is that many types of stem cells are likely to yield scientific advances and potentially new therapies. and it would be foolish to place all of our bets on certain stem cells at such an early stage in the development
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of this field. for this reason the international society for stem cell research has repeatedly recommended that all forms of stem cell research must be pursued and that patients should be cautious about claims regarding unproven adult stem cell therapies that are offered overseas. where would we be right now if you had taken the advice of opponents of embryonic stem cell research and directed the national institutes of health to focus their funding on umbilical cord blood cells or amniotic cells? promising research would be abandoned in favor of the alternatives, sacrificing scientific progress and the opportunity to develop new therapies. the award my lab received from president bush was for our work studying stem cells to give rise to the central nervous system. one of the things we discovered is that a birth defect is caused by defects in the function of these nurel stem cells during fetal development. in kids with this disease, the neweral stem cells failed to migrate into the intestine --
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part of the intestine, rendering that segment of the intestine nonfunctional. our work suggested that we might be able to bypass that defect by transplanting neweral stem cells into the nonfunctional portion of the t. the problem is that those cells only exist during fetal development. so we decided to generate those cells by deriving them from human embryonic stem cells. i want to emphasize this point, because for the therapy we want to use a tissue-specific stem cell, a cell tt in the newspaper is generally referred to as an adult stem cell and yet, we have to obtain it from embryonic stem cells. so this illustrates why it's scientifically meaningless to frame this debate as a choice between adult and embryonic stem cells, because we sometimes need embryic stem cells to derive the adu cells that we want to use in the therapy. so this research in my lab is funded by the national institutes of health, but it's suffered from repeated delays. first the grant was delayed while n.i.h. put in place its
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new embryonic stem cell policy. then we received the grant but unable to spend the money until n.i.h. had the opportunity to review new stem cell lines for funding. and finally, we were able to start the research but eight months later the federal injunction was issued. in the first few days after the injunction i told my lab if our funding was cut off, we would abandon our work on that disease. i have with me today jack mosher from my laboratory -- jack, you might want to stan up for a second. jack is the guy in my lab who does this work. the project i've been telling you about is jack's work and his salary comes exclusively from this grant. jack has dedicated the last nine years of h life to studying peripheral nervous system development culminating in this project, attempting to translate the basic science that we've done to the benefit of paties. yet, in those early days after the injunction, jack didn't know whether his work would survive the injunction, whether he would still have a salary, or what would happen to his career.
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so i'll just sum up by saying that american science is the envy of the world because is a mery tock crease in which there's fierce competition to funds the best ideas. if we accept the principle that those who are not judge to have the best ideas can obtain judicial relief that blocks funding of the best ideas to allow the lesser ideas to compete, this wl erode the veryard of american competitiveness. we owe more to the patients suffering from incurable diseases. we owe it to them to support all forms of stem cell research so that no matr where the science leads and where the cures come from, we can follow the most promising avenues of discovery. so i would urge you to clarify the wicker amendment so that there can be no question regarding congress's intent to fund the most meritorious science. >> thank you very much, very morrison. and mr. mosher, thank you. i may even have a question for you when we get to the questions here. we turn now to dr. nelson, and
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welcome and please proceed. >> thank you very much, chairman harkin, for the opportunity to present this information today. i am a translational neuroscientist from wayne state university. and today i am -- there are two types of stem cells, embryonic stem cells from elm bee yoss and adult stem cells. today i'm going to talk about adult em cells. and we use the term adult stem cells to mean not just stem cells from adults, but also from children and umbilical cord from blood. and what you have to understand is that the first human adult stem cell was isolated in 1992. now, we do have a long history of doing bone marrow transplants which contain stem cells. but adult stem cells are
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actually, in terms of looking at other diseases and injuries, is a new field. and it was onlyn the late 1990st that we realized the potential for other diseases other than cancer and various blood disorders. and, yes, most of the federal funding does go to adult stem cells, but the majority of that goes to old, but very important studies, in terms of tating cancer and blood diseases. the biotech industry that has a much larger budget for research than federal funding is not interested, in most cases, in adult stem cells. and we only have a limited amount of federal fding available. and where we are in adult stem cells, i'm sure the members
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have seen previously that there are some examples of people being treated with adult stem cells where there is considerable improvement. but the research is going from isolated incidents, and i'm going to present clinical trial data in respectable journals where we need to move through clinical trials to standard of care. and to move from a basic science study is relatively inexpensive. several hundred thousand dollars. but for each clinical trial, you need billions of dollars. so we need a lot more federal support to move forward with adult stem cells. this is an example -- oops, i'm sorry. this is an example of one of the patients -- it didn't go
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on. ok. i'm sorry. >> well, this would have been an example of one of the patients that was treated, a quadriplegic that was treated and -- treated using a procedure that was developedn portugal by dr. carlos lima and his team. and this is a picture -- i think we do have a poster of this gentleman that i met several years ago, and he was treated with his own adult stem cell two years after his injury. and two years after his injury, this gentleman is now shown standing up, without anyone supporting him.
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he's not waving, but he was in the video, and with only braces on his -- only braces on his -- on a ft-ankle brace. so he is standing up, maintaining balance. and he can now walk with a walker. amazing. a quadriplegic walking with a walkerithout assistance. and this is the progress. but this isn't an isolated incident. if you look at the two publications that have been published in peer review journals -- >> doctor, can i interrupt for a quick question? >> yes. >> is he what would be referred to as atologous stem cells? >> yes, he was. and these stem cells were obtained from in his nasal -- from inside of his nose and used to treat his spinal cord
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injury. but this, as i said, isn't a isolated incident. there are peer review publications in a larger number of patients, and i would love the opportunity to bring this forward in the united states after completing a safety study. so patients don't have to go to other countries to have this done. another example -- this is doug rice, and he had several heart attacks and had chronic heart failure. and he was told in 2002 that he had two years to live. and he went to another country and had a treatment done, and he's alive and doing well. at the time he had the procedure done he could barely
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walk. but this is also not an isolated example. this is a published peer-reviewed article of a study where they used 191 patients, had adult stem cells, and compared to 200 patients with similar heart conditions. and the treated patients lived longer and also could zice more. now i have -- exercise more. now i have to move to a somewhat gross and i apologize for that. this is corneal blindness. this is the second leading cause for blindness in the country. on the left side it shows eyes of several surgeries that were unsuccessful and blind in that eye. but using stem cells, adult stem cells from their other guy, this shows the result several years later. and this particular one was 112 patients, and more than 75% of the time it was successful, and
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many of these patients regained normal vision in their eye. and let me just go to another example. so this is the study -- the published study that was published of these 112 patients in the new england journal of medicine. i'll go to one more patient. m showing these sort of poster patients or poster examples. but now they're supported by results from clinical trials. in the middle is joe dav. he had very severe sickle cell anemia and his parents were told that joe might not live to his teens. so he had the procedure using his brother's umbilical cord blood cells, and joe is absolutely doing fine right now and has no sickle cell
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symptoms. and there's been two published studies for sickle cell, which is a very painful condition. this first study, six out of seven patients no longer have sickle cell symptoms. and another study at -- this particular study was by n.i.h. scientists and published in the "new england journal of medicine" in 10 adults with sickle cell anemia. and most of the patients -- nine out of 10 of these patients no longer had sickle cell symptoms. the last patient that i'dike to show is barry gowdy, and he hadultiple sclerosis. and he went to northwestern memorial hospital and he had symptoms of m.s., and they have
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disappeared. he was part of a larger study that was published in a peer-review journal in lancet, and these patients showed significant functional improvement, and no one got worse in this degenerative disease. >> dr. nelson, i would like to ask you to wrap up. we have to get to the last speaker and we have some votes we have to get to. >> this is another study supporting that. i won't talk about the amazing results and newly diagnosed juvenile diabetes, but i'd just like to conclude that we need more federal funding. we need more n.i.h. funding so patients don't have to go to other countri and so these amazing results that i presented can go to clinical trials and become standard of care for u.s. patients that need their support. >> thankou very much, dr. pa doocy necessarily sofpblet and now, ms. unser, please proceed.
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>> thank you for allowing me to testify and use my voice on behalf of millions of americans living with debilitating diseases. i feel honored and frustrated as to why we are here today. 10 years ago my hero, my superman, christopher reeve, sat in his power wheelchair and using every breath he took chanks to a machine testified to congress with the hope that embryonic stem cell research would be federally funded. today, in 2010, we are still fighting for this promising and hopeful research to continue. embryonic stem cells are science based on hope, hope for improving the quality of life for millions of americans by providing better treatment and eventually cures. my journey began 11 years ago. i was a healthy 12-year-old kid who was active and had big dreams. everything changed on february 5, 999. i can't recall how it felt to put my feet on the floor, how i got dressed that morning or what i had for breakfast, but what i do remember is that in a
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matter of 20 minutes my body became paralyzed and my life drastically changed. i was playing basketball at school and suddenly couldn't catch my breath and my head started pounding with sharp pain. the school i was attending called the ambulance and while laying down in the locker room, my lg became numb and tingly. i picked it up, put it back down and couldn't feel the floor. i was scared out of my mind but i felt the doctors could fix it. it's an autoimmune disorder in which t immune system attacks the spinal cord, causing inflammation that damages the cells ta control sensory and movement of the body. after staying in the hospital for a couple of months, i went to rehabilitation where i learned how to do everything from a wheelchair. all the while having dreams of my feet imprinting in the sand. today i'm a 23-year-old woman who has learned to adapt to a life in a wheelchair and in a paralyzed body. even though i live life to the fullest and look as though i'm just sitting down in a wheelchair, i have to always
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worry about pressureores from constant sitting. i worry about my osteoporosis advancing in my bones for not standing and bearing weight, which led to a fracture of my leftemur. i worry about my scoliosis getting worse, curvature of the spin common with people with spinal cord injuries. i have advancing nerve pain and adder complications. i am one of millions of americans living in various diseases and conditis that no matter how hard we try affects how we live our lives. the first time hope actually went something to me and became sort of my religion was when i saw what human embryonic stem cells can do. a year after i became paralyzed, my doctor and stem croyle scientist,oug kerr, who was at johns hopkins at the time, showed me a mouse that was once paralyzed and now can bear its weightnd take steps. at that moment i realized that this is science i couldn't ignore and it gave me a feeling of hope i wanted to fight for. which brings me to another
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point. it's frustrating to hear critics of is research say this is a path we can't go down and adult stem cells hold just as much promi as embryonic stem cells do. science is the pursuit of discovery and possibility. we should explore every opportunity and not count anhing out because i can't wait. and i know millions of americans now and in the future can't wait. in christopher reeve's testimony in 2000 he said "to obstacle should stand in the way of responsible investigation of their possibility." i'm here today to remove yet another obstacle in the path of this research, this answer, this hope. the political debate over this research is forcing many of our brillit scientists to think twice about whether they should stay in this field. i know how dedicated and passionate they are about helping all of us find answers who are pained and suffering. if we keep dragging this debate back here to washington, in congress and in the courts, more and more scientists will either find a different search avenue or move to
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another country where they can pursue the promise that embryonic stem cells possess. once and for all i urge congress to pass unambiguous legislation that allows this research to move forward. i grew up around racetracks and my family has won the indianapolis 500 a total of nine times. the goal of every driver is to pass under the black and white checkered flag first. the meaning of the checkered flag is winning. right now i can see the flag waving for me to go by, but with this current court ruling i feel that i have been driving under a long yellow caution flag. today i came here to say that this research is real, promising and hopeful to me and to others as we want so much to take that checkered flag and win our battles over diseases that constantly challenge our quality of life. thank you very much. >> thank you, ms. unser. we now have two more votes. .
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>> human embryonictem cells work. in 1998. we have only had 12 years to work.
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they are related to bone marrow transplantation from the 1950's bu. we have had decades of work. in 1955, all the patients died. he went back to the laboratory to find out why he was not able to do just transfer it from one patient to the other. you have to match the immune systems. the first successful transplant from an unknown donor was 1969, years later. we app this criteria of abandoning greasers that has not had sucess in 10 or 12 years. the nine of them when that exist today. -- then none of them with fixes
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today. >> one line, you said cells are different in important ways. i understand you have a grant to examine this very issue. tell me why this research is so important? what are the future discoveries that can be spurred? >> it is a major question to compare it this new and exciting research against the embryonic stem cell. we have won such a grant. i am losing a lot of sleep over the future of that grant. when the intervention was in place, it is threatened. it was going tobe pulled. very promising projects where
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all at risk. what our research has shown is that where as our panel is to make these cels as close as possible as to the others despite our best efforts, and there are still some differences. understanding those differences are essential to understand how they will be taped and all our research project. add to return back to the embryonic state, and they remember where they came from. that can be it vantage. we are working with cells that are derived. eight your interested in treating the disease ora leader disease predict liver disease,
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the fact that the thing in this may athwart your research. it is so ignorant about how these new stem cells will function. we continue to depend on an rihanna extend cells. >> dr. collins, i see these people that have been cured. i've had people like that in my own office who had stem cell treatment in another country. they come in and opey testify that now they can walk. one person had heart problems. why do we make of all this? >> the examples are exciting. to see the potential that is here. he was sent to appear reduce
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said something that god gave this to is for derstanding whher our treatmenworks are not, blinding and recommendation. if we have not applied thoe standards, in the have to be skeptical. things hapen and had nothing to do with the intervention. >> a blinding? >> the patience and investigators cannot know whether that individual received the new treatment. brenda visitation, patients can randomize one of the other city do not have a bias. for all of this involves, and so an effort has been put through, we have to be a little bit concerned about whether it to the journal lies. -- has been generalized. the research has not yet reached
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that standard in terms of the spinal cord results. i think it is very exciting to see how that is. i'm not saying we should be supporting the research. but the clear about what we consider to be proof of research. >> i have to go vote. i will give you a minute. >> he is correct. what i am sayingis we need the funds
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there is a problem and that this is a surgical technique. they are actually putting the tissue into the spinal cord the bi.
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we need to move in as directions. it is expense. quite the thing funding should be prohibited? >> this gets -- i am here as a scientist and not as a giving a personal opinion. as a scientist, what i came here to say is that in this country is that we need funding said they can become standard of care. it is not toanyone in the go to treat five or 10 patients. >> i thank you very much for being here today. you ara courageous young woman.
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than you for moving ahead with it. i t
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now available in more than 1 million homes, provided by cable, as public service. >> "washington journal" is next. we'll take your calls. and later this morning, the family research council holds a day-long conference. speakers include mitt romney and delaware's nominee for senate, christine o'donnell. that's live at 10:00 eastern. and coming up this hour, democratic representative donna edwards joins us to talk aboutt her party's perspectives, and then david l